Ventus Therapeutics Awarded Grant by the U.S. Department of Defense to Study the Role of cGAS in Lupus

Michael Crackower, Ph.D.

WALTHAM, Mass. & MONTREAL– Ventus Therapeutics, Inc., a biopharmaceutical company utilizing structural biology and a proprietary computational platform to identify and develop small molecule therapeutics across a broad range of diseases, announced today that it has been awarded a Lupus Research Program Idea Award from the U.S. Department of Defense (DoD). The multi-year grant will be used to study the role of the DNA sensor, cyclic GMP-AMP synthase (cGAS), in lupus and the potential for treatment using a cGAS inhibitor.

“It is estimated that over five million people worldwide have a form of lupus, and Ventus’ cGAS inhibitor may provide a new treatment option that avoids the risks of broad immune suppression,” said Michael Crackower, Ph.D., Chief Scientific Officer of Ventus, who will serve as the principal investigator for the DoD grant. “We are honored to receive this grant from the DoD in recognition of our innovative research on the complex etiology of this disease and advancement of new potential treatments towards the clinic.”

Systemic Lupus Erythematosus (SLE) is a chronic multi-organ auto-inflammatory disease for which there remains a high unmet medical need. One of the many complications of SLE is hypersensitivity to sunlight, especially ultraviolet B (UVB) wavelengths. This susceptibility affects up to 70% of patients and can result in painful skin lesions that cause scarring, hair loss and discoloration. While the etiology of SLE has been linked to multiple genetic and environmental factors, aberrant accumulation of DNA and elevated levels of type I interferons are believed to play central roles in its pathophysiology. As a sensor of DNA, and an inducer of Type I IFNs, cGAS has emerged as a therapeutic target of high interest for SLE.

“It has been gratifying to work with the Ventus team using their unique technology to create small molecule inhibitors of cGAS,” said Thomas Tuschl, Ph.D., Professor of RNA Molecular Biology at The Rockefeller University and co-investigator for the DoD grant. “cGAS is a well-validated target that has been very challenging for drug discovery.”

“We are focused on meeting the desire of patients to have access to an oral or topical therapeutic that can control the symptoms of cutaneous lupus and improve their quality of life,” said Kelly Pike, Ph.D., lead biologist of the cGAS program and Head of Translational Sciences and Scientific Affairs at Ventus and co-investigator for the DoD grant. “Using our ReSOLVE platform, Ventus has solved the high-resolution co-crystal structure of cGAS in the presence of novel binding compounds, has identified novel, highly selective and optimized cGAS functional inhibitors, and is working to bring them to the clinic.”